Acceleron plans to launch Phase 2 FSHD trial this year

According to a Boston Business Journal story, Cambridge, Massachusetts-based biotech Acceleron Pharma has plans to test its ACE-083 compound in facioscapulohumeral muscular dystrophy patients in a phase 2 clinical trial later this year: “The company presented top-line data from a Phase 1 trial in healthy volunteers showing the drug, which is injected into the muscle, was able to increase the size of a patients thigh muscle by up to 14 percent versus placebo. CEO John Knopf described the results as “unprecedented” and said that the company plans to advance ACE-083 into a phase 2 trials in patients with facioscapulohumeral muscular dystrophy in mid-2016.”

Hard to say. Acceleron is planning a Phase 2 trial, which could take 1-2 years depending on how fast they can recruit participants. They then need to analyze the data and if it looks promising, make a case to the FDA to allow a Phase 3 trial, which is a larger study to prove efficacy. That could take an additional 1-2 years, and then they need to apply for FDA approval.

An increase of muscle mass doesn’t mean a gain in muscle strength…
It as been shown in trials on inhibition of myostatin if I remember well (I’m not sure…).
What’s your idea on that subject ?
Thank you in advance for your answer.
Sylvie, from AFM FSH group, France

There is a myostatin inhibitor trial that is further ahead. Some results available below: Keep track of the trial on the ppmd website. Yes, I know that it is being tested on DMD boys but I participated in a previous webinar and was assured it would also work on FSH.